This review discusses current bottlenecks in making CRISPR-Cas9-mediated genome editing a therapeutic reality and it outlines recent strategies that aim to overcome these hurdles as well as the scope of current clinical trials that pioneer the medical translation of CRISPR-Cas9. Additionally, this review outlines the specifics of disease-modifying gene editing in recessive versus dominant genetic diseases with the focus on genetic myopathies that are exemplified by Duchenne muscular dystrophy and myotonic dystrophies.
CITATION STYLE
Conboy, I., Murthy, N., Etienne, J., & Robinson, Z. (2018). Making gene editing a therapeutic reality. F1000Research. F1000 Research Ltd. https://doi.org/10.12688/f1000research.16106.1
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